One woman’s remarkable resolve to help give better treatment options for people with rare blood cancers

Publish Date: 2/3/2017

A better treatment option for people with the blood
cancer myeloproliferative
neoplasms (MPN), is one step closer to being made available to Australians,
with the new drug interferon alfa-2a (Pegasys®), being discussed at today’s Pharmaceutical Benefits Advisory Committee
(PBAC) meeting.

This is especially exciting news for Melbourne
woman, Nathalie Cook who has worked tirelessly behind the scenes over the last
six years campaigning for this drug to be made available to Australians.

Nathalie herself was diagnosed with the MPN called essential thrombocythaemia in 2008, which
progressed to another MPN, polycythaemia vera, in 2010.

Seeking
more information about her diagnosis and treatment, she attended the MPN
doctor-patient conference at the Mayo
Clinic in the U.S. in 2011 where she hoped to gain information to help
her decide whether to continue with
her current treatment, or try a different drug, interferon alfa-2a (Roferon-A®)*.

Nathalie said the haematologists explained how
interferon can cause MPN patients to go into deep molecular remission.

She also learned that the newer form of interferon
– Pegasys – was easier to tolerate compared to Roferon, with fewer adverse
side-effects.

However Pegasys was not yet listed on the Pharmaceutical
Benefits Scheme (PBS).

“On the flight home, I made a decision – to try to
get Pegasys on the PBS for people with MPN in Australia.”

Whilst taking Roferon Nathalie experienced a number
of severe side-effects.

“I made several phone calls to the manufacturers of
Roferon and Pegasys, to report the adverse side-effects I experienced.

“I asked if they had plans to request the government
to add Pegasys to the PBS, but was told the company had no plans to make a
submission.

“In
early-2013, I had the opportunity to start treatment with Pegasys and the
side-effects were much milder. Within weeks my hair loss stopped and it began
to grow back, I had more energy, felt better and the flu-like symptoms
diminished.”

Nathalie attended the Mayo Clinic MPN conference
again in 2013 and 2015 and in this time continued to send annual letters to the
drug company Roche documenting her experience.

In 2015 Nathalie submitted a letter to the Senate
Enquiry on the Availability of New, Innovative and Specialist Cancer Drugs in
Australia, proposing the government include Pegasys on the PBS for the
treatment of MPN.

She also met with her local MP who suggested she
write to the Health Minister and also contacted Melbourne haematologist,
Associate Professor Constantine Tam, to ask about clinical trials.

“He told me the Australasian Leukaemia &
Lymphoma Group had previously requested drug company, Roche run a clinical
trial of Pegasys treatment in MPN patients in Australia, however they showed no
interest in doing so at that time,” she said.

Meeting with the
PBAC

In October 2016, at the Disrupting Cancer Forum in
Canberra, Nathalie met Professor Andrew Wilson, Chair of the PBAC.

“I spoke to him about the issue and he listened to my
story with great interest. I also asked if I could write to him directly.”

In mid-January 2017, she received a response from
the Department of Health to her letter to the Health Minister, advising that
her submission to Prof. Wilson was being considered at the March 8 meeting of
the PBAC.

“I was really pleased, delighted,” said Nathalie.

The Leukeamia Foundation also prepared a supporting
PBAC submission.

“Nathalie’s
remarkable resolve has put Pegasys on the PBAC’s agenda. This is no small feat
and our combined advocacy must continue,” said General Manager, Research,
Advocacy & Services, Caroline Turnour.

Path to PBS listing

“The path to PBS listing isn’t straight forward in
this instance. Pegasys isn’t registered by the Therapeutic Goods Authority
(TGA) for MPN and this can only be done by a pharmaceutical company,” Caroline
said.

Only a pharmaceutical sponsor (in this case, Roche)
can seek TGA registration and PBS listing for any drug or particular
indication.

“We keenly await the outcome of this meeting and
are committed to working with the government and Roche to overcome any barriers
that may stop it being listed.”

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